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Antisense Oligonucleotide Targeting DMPK in Patients with Myotonic Dystrophy Type 1: a Multicentre, Randomised, Dose-Escalation, Placebo-Controlled, Phase 1/2a Trial

Charles A. Thornton,Richard Thomas Moxley,Katy Eichinger,Chad Heatwole,Laurence Mignon,W. David Arnold,Tetsuo Ashizawa,John W. Day,Gersham Dent,Matthew K. Tanner,Tina Duong,Ericka P. Greene,Laura Herbelin,Nicholas E. Johnson,Wendy King,John Kissel,Doris G. Leung,Donovan J. Lott,Daniel A. Norris,Evan M. Pucillo, Wendy Schell,Jeffrey M. Statland,Nikia Stinson,Sub H. Subramony,Shuting Xia,Kathie M. Bishop,C. Frank Bennett

The Lancet Neurology（2023）

Cited 14|Views48
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